Adelaide researchers are contributing to a world-first in childhood brain cancer.
Can you imagine anything more heartbreaking than a child having cancer?
And worse – cancer in their brain, a location that is very difficult to access and has close to no hope of being treated successfully.
It would be devastating, but thankfully local scientists are close to introducing a new therapy for childhood brain cancer in a world-first that would give hope to many kids and their families.
The Hospital Research Foundation (THRF) is proud to be funding the last stage of testing for this new breakthrough treatment, to ensure it is safe to be given to patients possibly as early as next year.
Dr Tessa Gargett, Research Officer at Adelaide’s Centre for Cancer Biology (an alliance between the University of South Australia and SA Pathology), is leading the work which involves the use of science’s latest development: CAR T-cell therapy.
CAR T-cell therapy involves using a patient’s own cancer-fighting T-cells, genetically engineering them in the lab with Chimeric Antigen Receptors (CAR), and then returning them to the patient in the hope they can attack the cancer.
“There’s a really rare brain cancer called diffuse midline glioma that only effects kids,” Dr Gargett explains.
“It is inoperable because of where it’s located, it’s right in the middle of their brain so they can’t have surgery. They can have some radiotherapy, but it doesn’t really prolong their life at all. It is sadly a death sentence for these kids.
“They often get diagnosed about two or three years old, which is really devastating for their families. A lot of the parents and carers say that the thing that upsets them the most is that they don’t have a lot of treatment options.
“So we’ve had to come up with a trial that would be something new, something that hasn’t been tested before and that will bring hope to these families.”
CAR T-cell therapy has shown to be successful in children with leukaemia, and research teams globally are now seeing if it will work in other types of cancer such as melanoma and breast cancer. Dr Gargett and her collaborators in Sydney and Stanford Medicine in the US are one of the only groups trying it in childhood brain cancer.
“We can genetically engineer a patient’s own white blood cells, which are responsible for fighting off infection. We do this by introducing some new DNA, and that allows them to recognise the tumour cells,” she continues.
“The idea is that they find their way into the brain, find the tumour cells, bind to them and start killing them.
“The challenge is we need them to only attack the tumour cells and not the healthy brain cells. But our initial safety testing in the lab looks good, they are distinguishing between a tumour cell and say a neurone, so that’s given us the confidence to go forward.”
The last stage of pre-clinical testing is being undertaken with funds from THRF – a crucial step in order to gain approval from the Therapeutic Goods Administration to run clinical trials with patients at Sydney Children’s Hospital, where Dr Gargett is collaborating.
The Senior Paediatric Oncologist at Sydney Children’s Hospital, Associate Professor David Ziegler, this month secured a $1.1 million grant from the Federal Government to run these world-first clinical trials, once ready.
“We are so grateful to THRF supporters who have enabled us to receive this grant – we see this as bridging funding to help us get over this last hurdle to clinical trials,” says Dr Gargett.
“To nearly be at the stage where we can take our work in the lab and apply it to real patients, that’s very rewarding.”